The course aims to provide the skills needed to understand the methods for the generation, validation and commercialization of advanced therapy medicinal products (ATMPs), according to the definitions and guidelines established by drug regulatory agencies (EMA and FDA). The course therefore represents an advanced in‑depth study of pharmacology concepts applied to specific case studies.
Course Prerequisites
A strong background in cell biology, molecular biology and pharmacology is highly recommended.
Teaching Methods
The course will combine lectures and case study presentations, as well as guest seminars led by industry experts. Additionally, an integral part of the course will involve study groups coordinated by the instructor, designed to help students analyze an EMA assessment report to extract key data on the quality, efficacy, and safety of an ATMP.
Assessment Methods
The exam is oral and will cover the topics presented during the course, in order to verify mastery of the specific terminology and knowledge of the molecular aspects and reference guidelines for the design and preclinical and clinical validation of an ATMP.
Texts
The teaching material consists of the slides presented by the instructor during the lectures, supplemented by scientific publications and official EMA assessment reports relating to the ATMP examples discussed in class.
Contents
The course represents an advanced in‑depth study of pharmacology concepts applied to specific case studies. First, the different types of ATMPs will be classified, with a specific focus on genetic and tissue engineering techniques. Some examples of commercially available ATMPs will then be presented, retracing the process that led first to the identification of the molecular target, and subsequently to the development and preclinical validation of the therapeutic approach (pharmacokinetics, pharmacodynamics, and toxicology aspects), and finally to the clinical trial pathway.
Course Language
English
More information
The slides shown at classes will be provided to the students. Moreover, when necessary, the professors will provide to the students the integrative material that is most suitable for the preparation for the exam.
